One of the world's top ex­perts in coro­nary heart dis­ease is spear­head­ing a new gene edit­ing up­start out to trans­form the field

As head of the Cen­ter for Hu­man Ge­net­ic Re­search at Mass­a­chu­setts Gen­er­al Hos­pi­tal and the Broad’s Car­dio­vas­cu­lar Dis­ease Ini­tia­tive, Sekar Kathire­san has oc­cu­pied a sin­gu­lar po­si­tion as one of the world’s lead­ing ex­perts on the con­nec­tion be­tween ge­net­ics and coro­nary heart dis­ease. He’s tracked how peo­ple dealt a bad ge­net­ic hand — and the el­e­vat­ed risks that come with it — can lim­it in­her­ent dan­gers by lifestyle changes, and pon­dered over the ef­fects of dai­ly drugs used to treat mass pa­tient groups. And he’s reached a sim­ple con­clu­sion:

None of it is re­al­ly work­ing. 

In par­tic­u­lar, none of that is any­where near as use­ful as the ge­net­ic mu­ta­tions that he’s seen that con­fer a low­er risk of dy­ing and car­dio events. Par­tic­u­lar­ly the in­di­vid­u­als who car­ry mu­ta­tions “which break ei­ther of two genes — APOC3 or ANGPTL3 — rapid­ly clear triglyc­eride-rich lipopro­teins from the cir­cu­la­tion” and pro­tect them from heart at­tack.

Now, he’s mak­ing a pro­fes­sion­al leap to see if he and a squad of in­ves­ti­ga­tors at a new­ly launched biotech can dra­mat­i­cal­ly change the im­per­fect sta­tus quo through gene edit­ing.

“Imag­ine,” he tells me, “an in­jec­tion ad­min­is­tered once in life that safe­ly con­fers last­ing pro­tec­tion.”

No more pills. No fleet­ing com­mit­ments to healthy lifestyles that can’t stretch past Jan­u­ary. But a wide­spread and durable shar­ing of the same health ben­e­fits he’s seen in the very, very few. That’s the dream.

To­day, Kathire­san is step­ping down from his lofty aca­d­e­m­ic roles and mak­ing his de­but as CEO of Verve Ther­a­peu­tics, which is tak­ing its place in the hotbed of gene ther­a­py work around Cam­bridge, MA. The small team of 10 — soon to dou­ble in size — may not come close to ri­val­ing the big biotechs that oc­cu­py the streets in and around Har­vard and MIT. But rel­a­tive­ly few can claim the same kinds of con­nec­tions in the realms of drug sci­ence.

Ki­ran Musunuru

An­oth­er car­dio ge­net­ics ex­pert, Penn’s Ki­ran Musunuru and Har­vard pro­fes­sor J Kei­th Joung, who co-found­ed gene ther­a­py pi­o­neer Ed­i­tas, are on board as sci­en­tif­ic co-founders. There’s an al­liance with Beam Ther­a­peu­tics, the up­start next-gen gene edit­ing out­fit found­ed by Feng Zhang, one of 3 sci­en­tists wide­ly cred­it­ed with ush­er­ing in the CRISPR rev­o­lu­tion that has armed re­searchers around the world with ef­fec­tive tools to ac­com­plish their work. And Ver­i­ly will help work on the nanopar­ti­cles they plan to use for de­liv­ery.

Burt Adel­man, the for­mer EVP of R&D at Bio­gen, will chair the board, which in­cludes the Broad’s chief da­ta of­fi­cer, An­tho­ny Philip­pakis.

Beam will pro­vide some of the tech, and has an op­tion to step in on fu­ture com­mer­cial­iza­tion. Verve has al­so nailed down CRISPR patents, in­clud­ing Cas9 and Cas12a (Cpf1), from the Broad In­sti­tute and Har­vard Uni­ver­si­ty.

And they have $58.5 mil­lion in cash to do their work from GV (you prob­a­bly still think of them as Google Ven­tures), which is step­ping in with ARCH Ven­ture Part­ners, F-Prime Cap­i­tal, and Bio­mat­ics Cap­i­tal to form the orig­i­nal syn­di­cate.

To be suc­cess­ful, the Verve team un­der Kathire­san will not just have to demon­strate their ap­proach can safe­ly work, but al­so that it ul­ti­mate­ly can be done on a mass ba­sis in eco­nom­ic terms. That’s a tall or­der, but they do have some ad­van­tages, per­haps most no­tice­ably the ad­vances the ground­break­ers have made at the FDA, says the sci­en­tist.

“Gene edit­ing has the po­ten­tial to com­plete­ly trans­form the treat­ment par­a­digm for the dis­ease,” says Musunuru. “Pre­clin­i­cal stud­ies con­duct­ed in the field, in­clud­ing work done in my lab, have shown the promise of gene edit­ing to safe­ly re­duce cho­les­terol and oth­er coro­nary artery dis­ease risk fac­tors.”

So far, gene edit­ing in the le­git­i­mate R&D world has been cen­tered on the painstak­ing ad­vances of a hand­ful of pro­grams aimed at rare dis­eases. And Verve will start with its own rare ail­ments, tar­get­ing pa­tient pop­u­la­tions with the high­est un­met med­ical need. But Kathire­san isn’t drop­ping his pres­ti­gious aca­d­e­m­ic roles to search for mar­gin­al gains. He wants to tack­le the whole threat on a world­wide ba­sis.

That, of­fi­cial­ly, starts at Verve to­day.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Paul Tesar (Convelo Therapeutics)

Io­n­is, lead­ing MS re­searcher throw an­ti­sense at a new type of brain cells

No matter how many molecules he threw at them, Paul Tesar couldn’t get the brain cells to survive. Or he got them to survive, but then — to everyone’s bafflement — they still couldn’t do what they were supposed to.

Tesar, a professor of innovative therapeutics at Case Western University, had spent years building stem cell models for multiple sclerosis, growing brain organoids in dishes and then seeing what small molecules restored myelin production. Now he was trying to do the same for other myelin diseases, particularly an ultra-rare genetic condition called Pelizaeus-Merzbacher disease, where a single mutation leads to the death of the myelin-producing neurons, called oligodendrocytes, and can kill patients in infancy.

Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

Covid-19 roundup: Vac­cines will need to beat place­bo by 50% to qual­i­fy for FDA OK; UK tri­al drops Kale­tra

The FDA will set the bar for approving a Covid-19 vaccine at 50% efficacy, the Wall Street Journal reported, meaning any successful candidate will have to reduce the risk of coronavirus disease by at least half compared to placebo.

That requirement is part of guidance that the agency is set to release later today, laying out detailed criteria for vaccine developers — some of whom are eyeing an OK by the end of the year, in line with expectations at Operation Warp Speed.

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